Research ArticleNeurodegenerative Disease

An miRNA-mediated therapy for SCA6 blocks IRES-driven translation of the CACNA1A second cistron

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Science Translational Medicine  13 Jul 2016:
Vol. 8, Issue 347, pp. 347ra94
DOI: 10.1126/scitranslmed.aaf5660

Blunting the sharp end of the spear

Selectively turning off disease genes without disrupting other processes has been a growing goal of genetic research. In a new study, Miyazaki et al. work with a gene that expresses two proteins, a calcium channel, necessary for life, and a regulatory protein, α1ACT, which when mutated causes a form of ataxia called SCA6. These investigators figured out how to block expression of the disease protein without affecting the calcium channel using a small sequence of RNA called miRNA. They then used a viral vector to deliver this miRNA to mice engineered to develop a severe form of SCA6 and successfully prevented the disease.

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